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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 13, 2017
Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice
Eric R Pozsgai, Danielle A Griffin, Kristin N Heller, et al.
Biology Open
|
September 6, 2023
Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Human Gene Therapy
|
December 22, 2020
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy
Danielle A Griffin, Eric R Pozsgai, Kristin N Heller, et al.
Human Gene Therapy
|
January 5, 2021
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Therapeutic Advances in Neurological Disorders
|
January 30, 2023
Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy
Natalie L Goedeker, Sachi D Dharia, Danielle A Griffin, et al.
Molecular Therapy. Methods & Clinical Development
|
March 31, 2022
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations
Jerry R Mendell, Anne M Connolly, Kelly J Lehman, et al.
Human Molecular Genetics
|
February 26, 2016
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy
Danielle A Griffin, Ryan W Johnson, Jarred M Whitlock, et al.
Molecular Therapy. Methods & Clinical Development
|
February 2, 2018
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD
Lindsay M Wallace, Nizar Y Saad, Nettie K Pyne, et al.
Molecular Therapy. Methods & Clinical Development
|
February 23, 2023
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model
Young-Eun Seo, Stephen H Baine, Amber N Kempton, et al.
Annals of Clinical and Translational Neurology
|
March 28, 2015
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models
Patricia C Sondergaard, Danielle A Griffin, Eric R Pozsgai, et al.
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Search research articles
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Showing results (1-10 of 20) with videos related to
Sort By:
Page
of 2
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 13, 2017
Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice
Eric R Pozsgai, Danielle A Griffin, Kristin N Heller, et al.
Biology Open
|
September 6, 2023
Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Human Gene Therapy
|
December 22, 2020
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy
Danielle A Griffin, Eric R Pozsgai, Kristin N Heller, et al.
Human Gene Therapy
|
January 5, 2021
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Therapeutic Advances in Neurological Disorders
|
January 30, 2023
Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy
Natalie L Goedeker, Sachi D Dharia, Danielle A Griffin, et al.
Molecular Therapy. Methods & Clinical Development
|
March 31, 2022
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations
Jerry R Mendell, Anne M Connolly, Kelly J Lehman, et al.
Human Molecular Genetics
|
February 26, 2016
Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy
Danielle A Griffin, Ryan W Johnson, Jarred M Whitlock, et al.
Molecular Therapy. Methods & Clinical Development
|
February 2, 2018
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD
Lindsay M Wallace, Nizar Y Saad, Nettie K Pyne, et al.
Molecular Therapy. Methods & Clinical Development
|
February 23, 2023
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model
Young-Eun Seo, Stephen H Baine, Amber N Kempton, et al.
Annals of Clinical and Translational Neurology
|
March 28, 2015
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models
Patricia C Sondergaard, Danielle A Griffin, Eric R Pozsgai, et al.
Page
of 2